LT3191139T - Pigmentiniam retinitui skirta rpgr genų terapija - Google Patents

Pigmentiniam retinitui skirta rpgr genų terapija

Info

Publication number
LT3191139T
LT3191139T LTEP15825383.1T LT15825383T LT3191139T LT 3191139 T LT3191139 T LT 3191139T LT 15825383 T LT15825383 T LT 15825383T LT 3191139 T LT3191139 T LT 3191139T
Authority
LT
Lithuania
Prior art keywords
gene therapy
retinitis pigmentosa
rpgr gene
rpgr
pigmentosa
Prior art date
Application number
LTEP15825383.1T
Other languages
English (en)
Inventor
Michael A. Sandberg
Basil PAWLYK
Tiansen Li
Xinhua SHU
Alan Finlay WRIGHT
Robin Ali
Original Assignee
Massachusetts Eye & Ear Infirmary
Ucl Business Ltd
The Government of the United States of America as represented by the Secretary, Department of Health and Human Service
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Massachusetts Eye & Ear Infirmary, Ucl Business Ltd, The Government of the United States of America as represented by the Secretary, Department of Health and Human Service filed Critical Massachusetts Eye & Ear Infirmary
Publication of LT3191139T publication Critical patent/LT3191139T/lt

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Saccharide Compounds (AREA)
  • Compounds Of Unknown Constitution (AREA)
LTEP15825383.1T 2014-07-24 2015-07-17 Pigmentiniam retinitui skirta rpgr genų terapija LT3191139T (lt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462028638P 2014-07-24 2014-07-24
PCT/US2015/040866 WO2016014353A1 (en) 2014-07-24 2015-07-17 Rpgr gene therapy for retinitis pigmentosa

Publications (1)

Publication Number Publication Date
LT3191139T true LT3191139T (lt) 2021-01-25

Family

ID=55163577

Family Applications (2)

Application Number Title Priority Date Filing Date
LTEP15825383.1T LT3191139T (lt) 2014-07-24 2015-07-17 Pigmentiniam retinitui skirta rpgr genų terapija
LTEP20199208.8T LT3821912T (lt) 2014-07-24 2015-07-17 Rpgr genų terapija pigmentiniam retinitui gydyti

Family Applications After (1)

Application Number Title Priority Date Filing Date
LTEP20199208.8T LT3821912T (lt) 2014-07-24 2015-07-17 Rpgr genų terapija pigmentiniam retinitui gydyti

Country Status (17)

Country Link
US (2) US10314924B2 (lt)
EP (2) EP3821912B1 (lt)
JP (3) JP6654760B2 (lt)
CN (1) CN107206105A (lt)
CA (1) CA2991750C (lt)
CY (1) CY1123793T1 (lt)
DK (2) DK3821912T3 (lt)
ES (1) ES2834402T3 (lt)
FI (1) FI3821912T3 (lt)
HR (1) HRP20202023T1 (lt)
HU (1) HUE052781T2 (lt)
LT (2) LT3191139T (lt)
PL (1) PL3191139T3 (lt)
PT (2) PT3821912T (lt)
RS (1) RS61307B1 (lt)
SI (1) SI3191139T1 (lt)
WO (1) WO2016014353A1 (lt)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6654760B2 (ja) 2014-07-24 2020-02-26 マサチューセッツ・アイ・アンド・イア・インファーマリー 網膜色素変性症のためのｒｐｇｒ遺伝子治療
US20190054117A1 (en) * 2014-12-19 2019-02-21 Novartis Ag Dimerization switches and uses thereof
WO2016145345A1 (en) * 2015-03-11 2016-09-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 and rpgr vectors for treating x-linked retinitis pigmentosa
GB201516066D0 (en) 2015-09-10 2015-10-28 Young & Co Llp D Treatment of retinitis pigmentosa
GB201704192D0 (en) 2017-03-16 2017-05-03 Nightstarx Ltd Treatment of Retinitis Pigmentosa
WO2019077159A1 (en) * 2017-10-20 2019-04-25 INSERM (Institut National de la Santé et de la Recherche Médicale) METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR
EP4364803A3 (en) * 2017-11-15 2024-06-05 The Regents of the University of Michigan Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
AU2021236233A1 (en) * 2020-03-11 2022-09-22 Massachusetts Eye And Ear Infirmary Gene therapy for NMNAT1-associated retinal degeneration

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4980286A (en) 1985-07-05 1990-12-25 Whitehead Institute For Biomedical Research In vivo introduction and expression of foreign genetic material in epithelial cells
EP0228458B2 (en) 1985-07-05 1997-10-22 Whitehead Institute For Biomedical Research Epithelial cells expressing foreign genetic material
JPS6259797A (ja) * 1985-09-10 1987-03-16 日本電信電話株式会社 地中管体の推進方法
DE3852823T2 (de) 1987-09-11 1995-05-24 Hughes Howard Med Inst Transduktionsveränderte fibroblasten und ihre anwendung.
WO1989005345A1 (en) 1987-12-11 1989-06-15 Whitehead Institute For Biomedical Research Genetic modification of endothelial cells
DE68927996T2 (de) 1988-02-05 1997-12-04 Hughes Howard Med Inst Modifizierte hepatozyten und deren anwendung
DK0556345T3 (da) 1990-10-31 1997-06-16 Whitehead Biomedical Inst Retrovirale vektorer,som er egnede til genterapi
US5767079A (en) * 1992-07-08 1998-06-16 Celtrix Pharmaceuticals, Inc. Method of treating ophthalmic disorders using TGF -β
WO2001038578A1 (en) 1999-11-24 2001-05-31 Cornell Research Foundation, Inc. Inherited retinal diseases at the canine rp3 locus: linkage, marker- and mutation-based tests
GB0008801D0 (en) 2000-04-10 2000-05-31 Medical Res Council Sequences
CN102925445A (zh) * 2011-08-08 2013-02-13 刘军 一种获得rpgr 基因新的转录剪切形式的方法
AU2013287281B2 (en) * 2012-07-11 2018-04-26 The Trustees Of The University Of Pennsylvania AAV-mediated gene therapy for RPGR x-linked retinal degeneration
GB201412011D0 (en) 2014-07-04 2014-08-20 Ucl Business Plc Treatments
JP6654760B2 (ja) * 2014-07-24 2020-02-26 マサチューセッツ・アイ・アンド・イア・インファーマリー 網膜色素変性症のためのｒｐｇｒ遺伝子治療

Also Published As

Publication number Publication date
US20200215203A1 (en) 2020-07-09
US20170216454A1 (en) 2017-08-03
CY1123793T1 (el) 2022-05-27
EP3191139A1 (en) 2017-07-19
FI3821912T3 (fi) 2024-08-05
PL3191139T3 (pl) 2021-07-05
PT3821912T (pt) 2024-08-01
US11045558B2 (en) 2021-06-29
RS61307B1 (sr) 2021-02-26
HUE052781T2 (hu) 2021-05-28
LT3821912T (lt) 2024-09-25
JP7198329B2 (ja) 2022-12-28
JP2022009333A (ja) 2022-01-14
JP2017523239A (ja) 2017-08-17
US10314924B2 (en) 2019-06-11
EP3821912B1 (en) 2024-06-12
WO2016014353A1 (en) 2016-01-28
CA2991750A1 (en) 2016-01-28
JP6966532B2 (ja) 2021-11-17
EP3191139A4 (en) 2018-01-10
PT3191139T (pt) 2020-11-05
JP2020073536A (ja) 2020-05-14
DK3821912T3 (da) 2024-08-05
DK3191139T3 (da) 2020-11-16
EP3191139B1 (en) 2020-10-07
HRP20202023T1 (hr) 2021-03-05
CN107206105A (zh) 2017-09-26
EP3821912A1 (en) 2021-05-19
CA2991750C (en) 2023-02-14
SI3191139T1 (sl) 2021-03-31
ES2834402T3 (es) 2021-06-17
JP6654760B2 (ja) 2020-02-26

Similar Documents

Publication Publication Date Title
IL284741B (en) Gene therapy for retinitis pigmentosa
IL284577A (en) Garden healing
LT3821912T (lt) Rpgr genų terapija pigmentiniam retinitui gydyti
GB201420139D0 (en) Factor IX gene therapy
GB201407322D0 (en) Gene therapy
GB201707212D0 (en) Gene therapy for ciliopathies
IL263085A (en) Garden healing
GB201513151D0 (en) Functional gene replacement therapy
GB201616821D0 (en) Gene therapy
GB201612104D0 (en) Gene therapy
GB201612105D0 (en) Gene therapy
SG10201912076UA (en) Gene therapy composition
GB201410595D0 (en) Factor IX gene therapy